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Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen

Marta Gómez-García de la Banda1,2,3, Alessandro Amaddeo4,5, Sonia Khirani4,5,6

  • 1Pediatric Neurology and ICU Department, AP-HP Université Paris Saclay, DMU Santé de l'Enfant et de l'Adolescent, Hôpital Raymond Poincaré, Garches, France.

Pediatric Pulmonology
|October 29, 2020
PubMed

Insights

Nusinersen treatment significantly improved respiratory muscle strength and lung function in children with Spinal Muscular Atrophy (SMA) Type 2. Motor function also showed improvement, offering new hope for SMA patients.

Area of Science:

  • Neurology
  • Pediatrics
  • Pulmonology

Background:

  • Spinal Muscular Atrophy (SMA) is a genetic disorder affecting motor neurons.
  • Nusinersen improves motor function in SMA but its effect on respiratory muscles is less understood.
  • Respiratory muscle weakness is a major concern in SMA patients.

Purpose of the Study:

  • To evaluate the impact of nusinersen on respiratory muscle performance and lung function in children with SMA Types 1c and 2.
  • To compare respiratory muscle strength in nusinersen-treated SMA Type 2 patients with historical controls.

Main Methods:

  • 16 children with SMA (2 Type 1c, 14 Type 2) received six nusinersen injections.
  • Respiratory muscle strength was assessed using maximal static inspiratory pressure, forced vital capacity, and esophageal pressure.
  • Motor function was evaluated using the Motor Function Measurement (MFM) and Hammersmith-Hine Scale (HINE-2) at baseline and 14 months.
  • Data from treated SMA Type 2 patients were compared to 14 age-matched historical controls.

Main Results:

  • Nusinersen-treated SMA Type 2 patients showed significantly improved inspiratory muscle strength compared to historical controls (p < .05).
  • Improvements were noted in maximal static inspiratory pressure, forced vital capacity, and esophageal pressure during maximal sniff.
  • Significant improvements in MFM and HINE-2 scores were observed in all 16 SMA patients after 14 months of nusinersen treatment.

Conclusions:

  • Nusinersen treatment leads to significant improvements in respiratory muscle performance in children with SMA Type 2.
  • The study demonstrates a positive effect of nusinersen on both respiratory and motor functions in pediatric SMA patients.
Abstract

Keywords:
lung functionmotor functionnusinersenrespiratory musclesspinal muscular atrophy

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